Placebo-controlled study in neuromyelitis optica : ethical and design considerations

Cree, B.A.C.; Bennett, J.L.; Sheehan, M.; Cohen, J.; Hartung, H.P.; Aktas, O.; Kim, H.J.; Paul, F.; Pittock, S.; Weinshenker, B.; Wingerchuk, D.; Fujihara, K.; Cutter, G.; Patra, K.; Flor, A.; Barron, G.; Madani, S.; Ratchford, J.N.; Katz, E.

Placebo-controlled study in neuromyelitis optica : ethical and design considerations

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Item Type:	Article
Title:	Placebo-controlled study in neuromyelitis optica : ethical and design considerations
Creators Name:	Cree, B.A.C., Bennett, J.L., Sheehan, M., Cohen, J., Hartung, H.P., Aktas, O., Kim, H.J., Paul, F., Pittock, S., Weinshenker, B., Wingerchuk, D., Fujihara, K., Cutter, G., Patra, K., Flor, A., Barron, G., Madani, S., Ratchford, J.N. and Katz, E.
Abstract:	BACKGROUND: To date, no treatment for neuromyelitis optica (NMO) has been granted regulatory approval, and no controlled clinical studies have been reported. OBJECTIVE: To design a placebo-controlled study in NMO that appropriately balances patient safety and clinical-scientific integrity. METHODS: We assessed the "standard of care" for NMO to establish the ethical framework for a placebo-controlled trial. We implemented measures that balance the need for scientific robustness while mitigating the risks associated with a placebo-controlled study. The medical or scientific community, patient organizations, and regulatory authorities were engaged early in discussions on this placebo-controlled study, and their input contributed to the final study design. RESULTS: The N-MOmentum study (NCT02200770) is a clinical trial that randomizes NMO patients to receive MEDI-551, a monoclonal antibody that depletes CD19+ B-cells, or placebo. The study design has received regulatory, ethical, clinical, and patient approval in over 100 clinical sites in more than 20 countries worldwide. CONCLUSION: The approach we took in the design of the N-MOmentum trial might serve as a roadmap for other rare severe diseases when there is no proven therapy and no established clinical development path.
Keywords:	Neuromyelitis Optica, Trial Design, Ethics, MEDI-551, Anti-CD19 Monoclonal Antibody
Source:	Multiple Sclerosis Journal
ISSN:	1352-4585
Publisher:	Sage Publications
Volume:	22
Number:	7
Page Range:	862-872
Date:	June 2016
Official Publication:	https://doi.org/10.1177/1352458515620934
PubMed:	View item in PubMed

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